Patent or Perish?

Like many researchers, I’ve always been motivated by the impact of science on medicine and health, and by the large number of people who can benefit from a single discovery. While academia can feel like a convoluted path for those of us interested in making an immediate impact on public health, the first step in translating your research is easier than you might think. So what is this key secret? I spoke with a principal investigator at MGH and entrepreneur, PhD Leileata Russo, about how to fast track our discoveries with the vision of moving them from the ‘bench-to-bedside’, and her answer was: Patents.

Dr. Russo is a scientific co-founder of a successful start-up, Exosome Diagnostics Inc., based upon a discovery made in an academic lab. After learning the ropes with her first start-up experience, she is now in the process of starting a second company.  Exosome Diagnostics Inc. is based upon the purification of exosomes from patient blood or urine for diagnostic purposes.  Exosomes are part of the extracellular microvesicle family.  They are shed by cells through the fusion of multi-vesicular bodies with the plasma membrane and they are thought to be involved in cell-cell communication.  Russo described the conception of the first company as a ‘light-bulb moment’.  She had heard about the discovery of RNA in exosomes from her lab-mate, PhD Kevin Miranda. The discovery had been made by PhD Johan Skog, another junior faculty member at MGH in the neurology department, who found that glioblastoma tumors release exosomes into the blood and that RNA within those exosomes reveal specific tumor point mutations (Skog, 2008). Russo and Miranda extended these findings to focus on urinary exosomes, and it was clear that the genitourinary system could be non-invasively transcriptionally profiled from a urine sample.  Russo recognized immediately that these findings had significant health impact for the diagnosis and monitoring of cancers.  Profiling specific mutations in cancers has become an important step in determining treatment.  Biopsies are the most common method used to identify tumor mutations but they are both invasive and can disregard the heterogeneity of a tumor. Exosomes keep RNA protected from degradation and therefore allow tumor mutations to be profiled from biofluids in a non-invasive manner. The work was quickly patented and Russo’s business connections were brought in to start the company Exosome Diagnostics, Inc.

Russo may have had a little more experience with the start-up and patent process than your average bench scientist; her PhD mentor at Monash University in Australia developed an FDA approved diagnostic tool, Accumin, which used high performance liquid chromatography (HPLC) to detect urinary albumin (a sign of kidney disease) earlier and with less false negatives than the immunobased assays that were currently used (Comper and Osicka 2005). This business connection was used to help get Exosome Diagnostics Inc funded and off the ground. An important take-away from Russo’s experience is the value of networking with people who have seen the start-up process from infancy through to an FDA-approved product and who have the right business connections.

The foremost advice Russo gives to academic researchers with an interest in moving their work from bench-to-bedside is to patent your findings.  Some academic researchers may view industry as the ‘dark side’ and that patenting their work could be considered a hindrance to the free use of their findings. However, this could not be further from the truth. Patenting a finding does not preclude its use in the research setting so you really have nothing to lose. There are also important reasons to patent your work. It can take hundreds of millions of dollars to bring a drug to market and before committing this type of capital, investors need to know that there is protection around the discovery that they are investing in. Furthermore, once there is a publication or even a published poster presentation concerning the finding that work is now in the public domain and is not ‘patentable’.  Therefore, Russo explained, you are actually patenting for the patient, not just to protect your own intellectual property.

To create a successful start-up you need an idea that 1) has a market 2) is able to be translated.  Academia gives researchers freedom to change their focus and explore new ideas as they arise but startups have many challenges such as time-dependent goals and milestones – it’s a “keep your eye on the prize” mentality. Startups offer many advantages such as networking opportunities and learning how to integrate research and business. Russo explained that creating a start up is “Overall … a challenging and exhilarating experience. It is like a rollercoaster ride – it has its peaks and troughs and at the end of the day you just want to get back on and experience it all again.”

So, while we have been indoctrinated with the ‘publish or perish’ mentality, if you want to see your findings translate from bench to bedside, take a minute before you submit that publication, and talk to your institution’s tech transfer office about submitting a patent application first.

 

Work Cited

Johan Skog, Tom Würdinger, Sjoerd van Rijn, Dimphna H. Meije, Laura Gainche William T. Curry, Jr., Bob S. Carter, Anna M. Krichevsky& Xandra O. Breakefield..  Glioblastoma microvesicles transport RNA and proteins that promote tumour growth and provide diagnostic biomarkers. Nature Cell Biology 10, 1470 – 1476 (2008)

Comper WD, Osicka TM. Detection of urinary albumin. Advances in Chronic Kidney Disease. 12(2),170. (2008)

Rachel Liberman

Rachel Liberman is postdoctoral research fellow at Massachusetts General Hospital where she works in the Nephrology division within the Program in Membrane Biology.  She is currently investigating the regulation of proteins involved in renal acid base homeostasis.  She received her PhD in Molecular Physiology from Tufts University Sackler School of Biomedical Sciences in 2014.  She is particularly interested in translational research and scientific communications.